Core Insights - Neurocrine Biosciences announced significant reductions in cognitive and motor-related burdens in adults with Huntington's disease chorea treated with INGREZZA (valbenazine) capsules, based on new patient-reported post-hoc data from the KINECT-HD study [1][4] Group 1: Study Findings - The KINECT-HD study is the first clinical trial to show measurable changes in cognitive and motor disease burden with a vesicular monoamine transporter 2 inhibitor in Huntington's disease patients [1][2] - In the 12-week Phase 3 KINECT-HD clinical trial, INGREZZA showed a significant reduction in chorea severity, with a placebo-adjusted mean reduction in the Total Maximal Chorea (TMC) score of 3.2 units (p<0.0001) [11] - Patient-reported analyses indicated greater reductions in cognitive and motor-related disease burden for INGREZZA compared to placebo across various domains, including memory loss and mobility limitations [3][4] Group 2: Cognitive and Motor-Related Improvements - Statistically significant improvements were observed in cognition-related items, such as memory loss (-0.9 for INGREZZA vs -0.2 for placebo) and decision-making abilities (-1.0 for INGREZZA vs -0.3 for placebo) [3] - For motor-related items, INGREZZA demonstrated larger reductions in scores compared to placebo, including abnormal movements (-1.5 for INGREZZA vs -0.8 for placebo) and impaired coordination (-1.0 for INGREZZA vs -0.4 for placebo) [3][5] Group 3: Background on Huntington's Disease - Huntington's disease is a hereditary progressive neurodegenerative disorder affecting approximately 41,000 adults in the U.S., characterized by motor, cognitive, and psychiatric symptoms [7] - Chorea, a common symptom of Huntington's disease, involves irregular and unpredictable movements that can interfere with motor coordination and daily activities [7] Group 4: About INGREZZA - INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved for treating chorea associated with Huntington's disease and tardive dyskinesia [14][15] - The drug is designed for once-daily administration without the need for titration, making it convenient for patients [16]

KINECT-PRO is the first and only study to specifically evaluate and demonstrate patient-reported improvement with vesicular monoamine transporter 2 inhibitor treatment on tardive dyskinesia using multiple clinically validated scales Patients taking INGREZZA reported robust and clinically meaningful improvements in physical, social and emotional functioning Improvements were seen as early as Week 4 after initial treatment with the lowest INGREZZA dose and sustained through Week 24 Even patients with milder ...

Core Insights - Neurocrine Biosciences announced significant improvements in symptoms and overall severity of schizophrenia in adults from the Phase 2 study of NBI-1117568, the first oral muscarinic M4 selective orthosteric agonist in clinical development for this condition [1][2][6] Company Overview - Neurocrine Biosciences is focused on developing treatments for neurological, neuroendocrine, and neuropsychiatric disorders, with a diverse portfolio that includes FDA-approved treatments and a robust pipeline of compounds in clinical development [13] Study Details - The Phase 2 study involved 210 adults aged 18 to 55 with schizophrenia, randomized to receive either NBI-1117568 or placebo over a six-week period, followed by a two-week safety follow-up [2][9] - The primary endpoint was the change in total Positive and Negative Syndrome Scale (PANSS) score from baseline to Week 6, showing statistically significant improvements with the 20 mg dose of NBI-1117568 by Week 3 [4][6] Safety and Tolerability - NBI-1117568 was generally safe and well tolerated, with treatment discontinuation rates due to adverse events similar to placebo; common adverse events included somnolence (10.7% vs 2.9%) and dizziness (9.3% vs 1.4%) [3][6] Future Development - Based on positive Phase 2 results, a Phase 3 registrational program has been initiated to further evaluate the efficacy, safety, and tolerability of NBI-1117568, expected to enroll approximately 280 patients [6][9]

Core Insights - Neurocrine Biosciences presented new analyses from a Phase 4 randomized withdrawal study showing that patients with tardive dyskinesia who continued treatment with INGREZZA® (valbenazine) capsules reported improvements in functional and health-related quality of life measures [1][2] - The findings were presented at the 2025 ISPOR Conference, highlighting the clinically meaningful improvements in patients' quality of life due to INGREZZA treatment [1][2] Study Details - The analyses were based on data from 127 patients in a Phase 4, double-blind, placebo-controlled study, where patients received up to 80 mg of INGREZZA for eight weeks before being randomized to continue treatment or receive a placebo for an additional eight weeks [2][3] - Significant improvements were observed in health-related quality of life (HRQoL) measures, including mobility, self-care, usual activities, and pain/discomfort, with placebo-adjusted differences noted in anxiety/depression and mobility [3][4] Health-Related Quality of Life Improvements - Patients receiving INGREZZA for eight weeks showed improvements in HRQoL dimensions, with changes from baseline reported as follows: mobility (-0.27), self-care (-0.28), usual activities (-0.36), and pain/discomfort (-0.34) [3] - Continued treatment for an additional eight weeks resulted in further improvements, including a placebo-adjusted difference in mobility of -0.34 and anxiety/depression of -0.38 compared to the placebo group [3] Functional Impairment Improvements - In the open-label portion of the study, patients reported significant improvements in work/school (-1.37), social life (-1.65), family/home life (-1.30), and total Sheehan Disability Scale (SDS) score (-4.28) after eight weeks of treatment [4] - Continued treatment for an additional eight weeks led to further improvements in social life (-0.95) and family/home life (-0.89) compared to those receiving placebo [4] About INGREZZA - INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved for treating adults with tardive dyskinesia and chorea associated with Huntington's disease [12][13] - The drug is designed to inhibit dopamine release, potentially reducing uncontrollable movements associated with these conditions [13][14]

Summary of Neurocrine's Conference Call Company Overview - **Company**: Neurocrine Biosciences - **CEO**: Kyle Gano, appointed in October 2023 Macro Issues - **Executive Order on Most Favored Nation**: - Uncertainty regarding the impact on Neurocrine due to lack of details on the executive order [3][4][5][6] - Focus on building a resilient business amidst evolving regulations [6][7] - **Tariffs**: - Tariffs are not expected to significantly impact the cost of goods for Neurocrine's products [9][10] - **FDA Interactions**: - No current NDA reviews; recent interactions focused on Phase II discussions for upcoming Phase III programs [11][12] Product Launch: Crinesity (Canestrafont) - **Sales Performance**: - Recognized $14.5 million in sales with over 400 new patient starts in the last quarter [16] - Impressive reimbursement rate of approximately 70% [16][18] - **Launch Strategy**: - The launch is described as "measured" due to the need for physician education and patient awareness [18][20] - Patients must navigate insurance processes, which may delay prescriptions [20][21] - **Patient Demographics**: - Initial patient population is skewed towards pediatrics due to more frequent physician visits [27][32] - Adult women are expected to show more motivation for treatment compared to men [49] - **Direct-to-Consumer Advertising**: - Traditional advertising may be less effective due to the smaller patient population for congenital adrenal hyperplasia (CAH) [34][36] - **Market Potential**: - Crinesity is viewed as a potential blockbuster, with expectations of significant market uptake [38][39] Competitive Landscape - **Differentiation**: - Neurocrine's approach with Crinesity is based on efficacy, safety, and tolerability, which are critical for patient adoption [43][46] - The company aims to treat the majority of patients with CAH before competitors enter the market [45][46] Pipeline Developments - **Upcoming Data Readouts**: - Phase III trials for osavapitur and MBI-568 are anticipated [52] - Studies on valbenazine for dyskinesia associated with cerebral palsy and adjunctive treatment for schizophrenia are ongoing [56][60] - **Muscarinic Agonists**: - Neurocrine is developing multiple muscarinic agonists, focusing on selective activation to improve efficacy and safety [70][72] Financial Outlook - **Investment in Growth**: - The company is prioritizing growth over immediate profitability, with significant investments in sales and marketing for Crinesity and INGREZZA [77][78] - R&D spending is projected to be in the mid-30% range of net revenue due to ongoing Phase III trials [79] Conclusion - Neurocrine is navigating a complex regulatory environment while launching Crinesity, which shows promising early sales. The company is focused on educating physicians and patients, with a strong pipeline and strategic investments aimed at long-term growth and market leadership in the treatment of CAH and other conditions.

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